Pivekimab Sunirine (PVEK) Shows Encouraging Results in Treating BPDCN

PVEK, a novel antibody drug conjugate, shows encouraging results in the treatment of BPDCN. 

Antibody drug conjugates in blood cancer

Antibody drug conjugates (ADCs) combine targeted therapy and chemotherapy to selectively target cancer cells with minimal damage to healthy tissue. For people with blood cancer, ADCs demonstrate encouraging results such as reduced side effects, improved treatment outcomes, and better overall quality of life.  

Several treatment options have been explored for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN), but more options are still needed to improve outcomes.  

In 2020, the U.S. Food and Drug Administration (FDA) granted pivekimab sunirine, also known as PVEK (AbbVie) breakthrough therapy status, which sped up its review as a treatment for relapsed/refractory BPDCN. PVEK works by directly targeting and delivering chemotherapy to cells with CD123, a protein that is overexpressed in BPDCN. 

PVEK Study Overview

At the 2025 American Society of Clinical Oncology (ASCO) meeting,  researchers presented findings from the phase I/II CADENZA clinical trial. This study analyzed how well PVEK improved response, survival, and allowed people to move to stem cell transplant, when used as a first-line therapy and in relapsed and refractory (R/R) BPDCN.  

The goal was to determine how well PVEK improved response, survival, and allowed people to move to stem cell transplant.  

There were 84 patients with CD123-positive BPDCN enrolled in this study. Of those, 31 received PVEK as a first-line therapy and 51 had received 1 to 3 treatments prior.   

How well PVEK worked as a first-line treatment

After five cycles of PVEK: 

• 70% of patients had a complete response (CR) or a CR with minimal skin changes for a median of 9.8 months. This means they had no detectable disease. 
• 85% of patients had a noticeable reduction in their cancer. This is the overall response rate (ORR).
• Half of participants were still alive 16.6 months after treatment.
• 39% moved on to stem cell transplant. Of those, 92% had no detectable cancer, and at the time of study analysis, overall survival had not yet been reached. 

How well PVEK worked for people with R/R BPDCN

After three cycles of PVEK: 

• 14% of people had a CR or a CR with minimal skin changes for a median 9.2 months after treatment.
• The ORR was 35%.
• Half of participants were still alive 5.8 months after treatment.
• 12% of patients moved on to stem cell transplant. 

Side effects and safety

When studying new treatments, it is just as important to understand side effects as it is to assess how well they work.  Side effects led to treatment being stopped in 9% of newly diagnosed individuals and 7% of those with R/R BPDCN.

There were no reports of treatment-related death or capillary leak syndrome, a serious complication where fluid leaks from blood vessels into surrounding tissue.  

Peripheral edema, swelling caused by fluid retention, was the most common side effect, occurring in 54% of people.  

2% of people developed veno-occlusive disease, a condition where some of the veins in the liver become blocked and decreased blood flow.  

Overall, PVEK offers meaningful benefits to patients, especially those newly diagnosed with BPDCN. Ongoing research will continue to clarify how safe and effective PVEK is in treating BPDCN. 

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Sources: 

• Efficacy and safety of pivekimab sunirine (PVEK) 
• Antibody-Drug Conjugates (ADCs) 
• Veno-occlusive disease 
• Capillary leak syndrome
• Breakthrough Therapy